“It’s one thing to ensure a treatment is available and it’s another thing to make sure that children can have equitable access to these critical treatments that can save their lives. We are launching #AADCmiles to ensure that no child is left behind when a cure is in reach even if they have to cross an ocean to reach it.”

Laura Hameed, Executive Director

Only 150 patients affected in the world

Aromatic Amino Acid Decarboxylase deficiency – AADC deficiency – commonly referred to as pediatric Parkinsonism, is a rare disease that affects the nervous system and has no cure. Gene therapy is a pioneering technique and a hopeful way to improve the quality of life of these children who can be treated in Poland or in the USA. Fundación Columbus and Columbus Children’s Foundation have partnered with gene therapy pioneer Dr. Krystof Bankiewicz and his team to advance and ensure as many children as possible can access these potentially life-saving treatments.

AADC is a rare genetic disorder that affects the brain’s ability to produce neurotransmitters, especially dopamine and serotonin. This causes the brain to be “disconnected” from the rest of the body and impairs speech and movement. These patients require continuous care and various drugs for treatment. But with gene therapy, many of the symptoms can be improved and the intake of these drugs can be reduced.

The Pioneer

Dr. Krystof Bankiewicz and his team are currently leading the Fundación Columbus’s health programme in Poland and the USA to improve the lives of patients with AADC deficiency through gene therapy. Almost 30 children have already been treated, as is the case of Irai, the girl who was “born” for the second time on 14 May 2019, at the Brodno Hospital in Warsaw. The operation was a success, as only twenty months after the operation, Irai started to walk.

Dr. Krystof Bankiewicz, CEO of Columbus Children’s Foundation, is a world-renowned expert in neurorestorative medicine, with successful translation of multiple gene therapies to the clinic. He holds the Kinetics Foundation Chair in Translational Research and is a resident professor of Neurological Surgery and Neurology at the University of California, San Francisco. He is also Vice Chair for Research in the Department of Neurosurgery and Director of the UCSF Center for Interventional Neurology.

Gene therapy treatments for AADC

Clinical trials based on gene delivery techniques used to treat children with Parkinson’s disease are being conducted at two US universities.


How can we help them?

Become a Gene Flight Sponsor.

Columbus Children’s Foundation and Fundación Columbus are teaming up to raise funds to add #AADCmiles and get transport children with rare diseases from around the world to the United States so they can receive gene therapy treatment.

You can fill up your tank by adding miles:

Irai: the girl who was born twice

Luke Cross, one of the children waiting to be “born again”.

Luke Cross is the only child in Ireland to be diagnosed with an extremely rare neurological condition called aromatic amino acid decarboxylase deficiency (AADC). Thanks to the tireless efforts of his mother, Emma Styles, Luke will have the opportunity to be treated in San Francisco by Dr Krystof Bankiewicz using gene therapy.

For Luke, the potential benefits of the new gene therapy surgery include reduced medication and improved muscle tone and an end to painful seizures. Luke’s day-to-day life is very hard for him and his family, as he needs round-the-clock care. He is completely dependent on everyone around him. He cannot walk or talk. He can’t eat anything by mouth, so he needs a lot of help.

Luke will travel from Dublin to the United States to access the gene therapy operation that could make it easier for him to take his first steps. 

#AADCmiles will help children just like Luke whether they are in Ireland or anywhere in the world access the care they need to give them a chance to live the lives they deserve.

Every donation to help a child with an ultra-rare genetic condition makes an immediate difference.

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